Haploidentical Stem Cell Transplant for Pediatric Blood Cancers
This study is looking at a new way to perform a type of stem cell transplant called a haploidentical hematopoietic stem cell transplantation (haplo-HSCT) for children and young adults up to 30 years old with certain blood cancers (pediatric hematologic malignancies). The study uses a special process called Ex Vivo T-cell receptor alpha-beta and CD19+ Depletion using CliniMACs Plus. This process helps prepare the donated stem cells to reduce risks. We want to see how safe this new transplant method is within the first 100 days and how well the new cells grow (engraftment), measured by blood counts recovering within 42 to 75 days after transplant. You might be able to join if you have specific types of B cell ALL that haven't responded well to previous treatments.
- Study design
- This is a single-arm pilot study, meaning all participants receive the same treatment. It aims to enroll 50 participants.
- What's involved
- Not specified in the trial record.
- Compensation
- Not stated in the trial record.
- Follow-up
- Safety will be measured for 100 days after transplant, and engraftment will be monitored for up to 75 days after transplant.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
NCT05011422
Haploidentical Hematopoietic Stem Cell Transplantation With Ex Vivo TCR Alpha/Beta and CD19 Depletion in Pediatric Hematologic Malignancies
Recruiting
PHASE1Up to 30InterventionalTreatmentWashington University School of MedicineInvestigator-initiated
~50 participants
Updated 2026-08-19 on ClinicalTrials.gov
What's tested:Ex Vivo T-cell receptor alpha-beta and CD19+ Depletion using CliniMACs Plus
At a glance
Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Safety as measured by the number of events occurring within the first 100 days post-transplant
Measured over Through 100 days post-transplant
+3 more outcomes measured
Conditions
Where it's being run
1 sites across 1 statesMissouri1
Study leadership
- Thomas Pfeiffer, M.D. · PRINCIPAL_INVESTIGATOR · Washington University School of Medicine
Who to contact
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Do you actually qualify for this trial?
Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.
Eligibility criteria
Inclusion
Must meet at least one of the following disease criteria:
B cell ALL in first remission and any of the following:
Persistent flow-based MRD at end-of-consolidation:
≥ 1% for NCI SR ALL
≥ 0.01% for NCI HR ALL
TCF3-HLF t(17;19)
KMT2A rearranged infant ALL, \< 6 months of age and presenting WBC of \> 300,000 or poor steroid response (peripheral blasts \>= 1000 /uL on day 8 of therapy
Other high-risk features not explicitly stated here, after discussion/approval with protocol PI.
B cell ALL in second remission and any of the following:
Early (\<36 months from start of therapy) marrow or combined relapse
Late (\>36 months from start of therapy) marrow or combined relapse with end-of re-induction flow MRD \>= 0.1%
Early isolated extramedullary relapse (\< 18 months from start of therapy)
Any B cell ALL in third or greater remission
T cell ALL in first remission
End-of consolidation MRD \> 0.1%
Any T cell ALL in second or greater remission
AML in first remission with any of the following high-risk features:
MRD ≥ 1% after first induction course
MRD ≥ 0.1% after second induction course
RPN1-MECOM
RUNX1-MECOM
NPM1-MLF1
DEK-NUP214
KAT6A-CREBBP (if \>= 90 days at diagnosis)
FUS-ERG
KMT2A-AFF1
KMT2A-AFDN
KMT2A-ABI1
KMT2A-MLLT1
11p15 rearrangement (NUP98 - any partner gene)
12p13.2 rearrangement (ETV6 - any partner gene)
Deletion 12p to include 12p13.2 (loss of ETV6)
Monosomy 5/Del(5q) to include 5q31 (loss of EGR1)
Monosomy 7
10p12.3 rearrangement (MLLT10b - any partner gene)
FLT3/ITD with allelic ratio \> 0.1%
RAM phenotype as evidenced by flow cytometry: bright CD56+, dim to negative CD45 and CD38 and lack of HLA-DR
Other high-risk features not explicitly stated here, after discussion/approval with protocol PI.
AML in second or greater remission
Mixed phenotype or undifferentiated leukemia in any CR
Secondary to therapy-associated leukemia in any CR
NK cell lineage leukemia in any CR
Myelodysplastic syndrome (MDS)
Juvenile myelomonocytic leukemia (JMML)
Available familial haploidentical donor.
Donor and recipient must be identical at a minimum of one allele of each of the following genetic loci: HLA-A, HLA-B, HLA-Cw, HLA-DRB1, and HLA-DQB1. A minimum of 5/10 match is required and will be considered sufficient evidence that the donor and recipient share one HLA haplotype.
No more than 30 years of age
Lansky or Karnofsky performance status \> 50%
Adequate organ function as defined below:
Cardiac: LVEF ≥ 40% at rest or SF ≥ 26%
Hepatic:
Total bilirubin \< 3 x IULN for age
AST(SGOT)/ALT(SGPT) \< 5 x IULN
Renal: GFR ≥ 60 mL/min/1.73m2 as estimated by updated Schwartz formula for ages 1-17 years (see Appendix B), 24-hour creatinine clearance, or renal scintigraphy. If GFR is abnormal for age based on updated Schwartz formula, accurate measurement should be obtained by either 24-hour creatinine clearance or renal scintigraphy. Renal function may also be estimated by serum creatinine based on age/gender. A minimum serum creatinine of 2x upper limit of normal is required for inclusion on this protocol.
Pulmonary:
O2 saturation ≥ 92% on room air without positive pressure support
FEV1, FVC, and DLCO ≥ 50% of predicted (for children unable to perform a pulmonary function test, a high-resolution CT chest may be obtained)
The effects of these treatments on the developing human fetus are unknown. For this reason, patients of childbearing potential must agree to use adequate contraception (hormonal or barrier method of birth control, abstinence) prior to study entry and for 24 months following transplant. Should a woman become pregnant or suspect she is pregnant while participating in this study, she must inform her treating physician immediately.
Ability to understand and willingness to sign an IRB approved written informed consent document (or that of legally authorized representative, if applicable).
Exclusion
Available matched related donor. A patient with a matched unrelated donor is eligible if urgent transplantation is required. A prior unrelated donor search is not required for enrollment.
Active non-hematologic malignancy. History of other malignancy is acceptable as long as therapy has been complete and there is no evidence of disease.
Currently receiving any other investigational agents at the time of transplant.
Active CNS or extramedullary disease. History of CNS or extramedullary disease now in remission is acceptable.
A history of allergic reactions attributed to compounds of similar chemical or biologic composition to conditioning agents used in the study.
Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection (bacterial, viral with clinical instability, or fungal), symptomatic congestive heart failure, or unstable cardiac arrhythmia.
Presence of significant anti-donor HLA antibodies per institutional standards. Anti-donor HLA Antibody Testing is defined as a positive crossmatch test of any titer (by complement dependent cytotoxicity or flow cytometric testing) or the mean fluorescence intensity (MFI) of any anti-donor HLA antibody by solid phase immunoassay.
Presence of a second major disorder deemed a contraindication for HSCT.
Pregnant and/or breastfeeding. Women of childbearing potential must have a negative pregnancy test within 14 days of the start of conditioning.
The preferred donor should be an adult aged at least 18 years or older. However, in circumstances where no suitable adult donor is available, consideration may be given to a minor donor aged 12 years or older. This exception only applies when all identified, otherwise eligible adult donors meet one or more of the following criteria:
Have a medical condition that poses unacceptable risk, including autoimmune disease, infection, hematologic disorder, malignancy, or a pathogenic germline mutation.
Have comorbidities that preclude safe administration of granulocyte colony-stimulating factor (G-CSF), placement of a pheresis catheter and/or a pathogenic germline mutation.
Served as a donor in prior haploidentical HCT.
Significant psychosocial or logistical barriers.
Meets the selection criteria as defined by the Foundation for the Accreditation of Hematopoietic Cell Therapy (FACT).
Able to understand and willing to sign an IRB-approved written informed consent document (or that of legally authorized representative, if applicable).
What this trial measures
- Safety as measured by the number of events occurring within the first 100 days post-transplantThrough 100 days post-transplant
-Events are death, disease recurrence or progression, and graft failure
- Engraftment as measured by time to neutrophil count recoveryFrom day of transplant (day 0) to 42 days (+/- 14 days) post transplant
Time to neutrophil recovery is defined as the first of 3 measurements on different days when the patient has an absolute neutrophil count of \>500/μL after conditioning.
- Engraftment as measured by time to platelet count recoveryFrom day of transplant (day 0) to 75 days (+/- 14 days) post transplant)
Time to platelet recovery is defined as the first day of a minimum of 3 measurements on different days that the patient has achieved a platelet count \> 50,000/μL AND did not receive a platelet transfusion in the previous 7 days. The exception is the case in which a patient receives platelet transfusions specifically to achieve a higher platelet threshold to allow for an invasive procedure or protection if determined to be at elevated bleeding risk.
- Donor cell chimerism as measured by short tandem repeat analysisThrough day +100
* Can use peripheral blood samples or bone marrow samples * The percent of donor-derived cells are sequentially followed.